Clinical Trial: A Study to Evaluate the Safety and Pharmacokinetics of RC001 in Children With Dravet Syndrome

U.S., July 1 -- ClinicalTrials.gov registry received information related to the study (NCT07675746) titled 'A Study to Evaluate the Safety and Pharmacokinetics of RC001 in Children With Dravet Syndrome' on June 16.

Brief Summary: This is an open-label, single-center study to evaluate the safety, tolerability, pharmacokinetics, and preliminary efficacy of intrathecal RC001 in patients with Dravet syndrome aged 2 to 18 years. The study includes a dose-escalation part followed by a fixed dose treatment part, with participant progression based on investigator-assessed safety and efficacy.

Study Start Date: Dec. 22, 2025

Study Type: INTERVENTIONAL

Condition: Dravet Syndrome (DS)

Intervention: DRUG: RC001 injection-Dose Escalation Cohort ...